Fair for Rare: Kalten’s Story

My son Kalten lives with the ultra-rare and debilitating disorder Morquio A or MPS IV.

The first and only treatment available for Australians with Morquio A, Vimizim® (elosulfase alfa), was considered by the Pharmaceutical Benefits Advisory Committee (PBAC) at its March 2016 meeting. The PBAC stated that the treatment cost too much, but accepted its clinical effectiveness. This outcome was made public in April this year and was sent to the Chief Medical Officer to approve funding for Vimizim under the Life Saving Drugs Program (LSDP) – which was set up to help people with rare diseases get access to important treatments. Yet, here we are nine months later and Vimizim still hasn’t been reimbursed by the Australian Government.

There are around 21 Australians with Morquio A, a genetic condition that affects major organs in the body. People born with Morquio A are missing, or don’t make enough of, an important enzyme and can’t break down certain materials in the body. This causes a number of serious health problems, with most children having to undergo dozens of surgeries before they reach the age of five.

Kalten was diagnosed with Morquio A in January 2015, just months after the Australian clinical trial of Vimizim closed. This meant we had no option but to wait for the Government to decide to fund Vimizim so that Kalten, and other patients who are newly diagnosed, can access the treatment. Besides Kalten, there were two other children who were also missing out on treatment because their diagnosis came after the clinical trial had closed.

Fortunately for Kalten and the two other Morquio children, the Sydney Children’s Hospital Network contacted the company that makes Vimizim, BioMarin, to ask them to help them children until the Government could approve funding through the LSDP. Thankfully, BioMarin agreed to provide all three children with compassionate access to Vimizim for three months.

The three month period was decided as it was thought this would be enough time for the Government to make its decision. But here we are again. On Friday 9 December, Kalten will have the last of his weekly infusions of Vimizim as part of the compassionate access agreement. After that, he will no longer have access to a treatment that is helping to slow the progression of his rare disease.

I know first-hand how this treatment has helped other children with Morquio A. One of Kalten’s friends, Beau Cosgrove from Taralga NSW, was lucky to access the Vimizim clinical trial. It’s been amazing to see how much Beau’s physical health and general wellbeing have improved since being on the treatment. He is able to move better, walk without help, breathe normally and start school – the improvement to his quality of life has been a joy for his family to see.

My only hope is that my son also is able to experience these types of benefits. But to do that, he needs to be given ongoing, long-term access to Vimizim. This can only be possible if the Government agrees to fund the treatment. Until then, all that lies ahead for my family is a future of uncertainty about what will happen to Kalten.

Children with ultra-rare diseases, like Kalten, should not have to wait months and months to find out if they will get access to important treatments that are developed to help them. Although the numbers of Morquio A patients are only small, it doesn’t make them any less deserving of receiving the best care and treatment.

Kalten was diagnosed with Morquio A in January 2015, just months after the Australian clinical trial of Vimizim closed. This meant we had no option but to wait for the Government to decide to fund Vimizim so that Kalten, and other patients who are newly diagnosed, can access the treatment. Besides Kalten, there were two other children who were also missing out on treatment because their diagnosis came after the clinical trial had closed.

Each day they wait for access to treatment is another day that their disease has to take its toll on their lives. On behalf of all Morquio families, and on behalf of all families of children with rare diseases, I ask the Minister for Health and the Australian Government to please make decisions on funding important treatments quickly. Our children don’t have time to wait.

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About Rare Voices Australia

Rare Voices Australia (RVA) is a national, not-for-profit organisation established in 2012 with a vision to be ‘the unified voice for ALL Australians living with a rare disease’.
RVA is Australia’s national alliance advocating for those who live with a rare disease.
RVA provides a strong common voice to promote health policy and a healthcare system that works for those with rare diseases.